NHS secures deal to trial lifesaving gene therapy

Zolgensma, labeled “the most expensive drug in the world” has been procured by NHS England. The landmark deal has been confirmed by NHS Chief Executive Simon Stevens, and the drug is said to be made available at an affordable price.  

The drug takes the form of gene therapy and is used to help infants with Spinal Muscular Atrophy; this condition can be fatal and causes paralysis, muscle weakness and progressive loss of movement. Infants born with severe type one SMA, which is the most common form of the disease have their life expectancy to be estimated at 2 years.  

Studies have found that Zolgensma, has helped babies sit up, regain movement, and breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment. 

NHS England Chief Executive Sir Simon Stevens said: “This deal is a life-changer for youngsters with this cruel disease and for their families. 

“Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers. 

“Although the health service is still under real pressure from covid, and NHS England is also focused on leading the national covid vaccination rollout, today’s agreement is an important reminder that the NHS is looking after millions of other patients too, for whom real medical advances are now possible.” 

Novartis Gene Therapies, who manufacture the drug, struck a deal with NHS England to order a shipment at a ‘discount’ and its now opens the door for NICE to publish draft guidance on Zolgensma and how to use it effectively.  

Zolgensma is used using a single intravenous injection and contains a replica of the missing gene SMN1.  The drug is the second treatment for SMA made available after the NHS approval of Spinraza, which was available for patients in May 2019.

An ongoing assessment is currently underway to find centres of excellence to deploy the treatment.